RPE65-related recessive retinopathy
MONDO:0100368A retinopathy, which may include conditions described as retinitis pigmentosa and Leber congenital amaurosis, caused by biallelic variants in the RPE65 gene.
Also known as: RPE65-related recessive retinopathy, recessive RPE65 retinopathy, LCA2, Leber congenital amaurosis 2, Leber congenital amaurosis caused by mutation in RPE65, Leber congenital amaurosis type 2, RP20, RPE65 Leber congenital amaurosis
33 clinical trials for this condition and its sub-types, 5 tagged with RPE65-related recessive retinopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of RPE65-related recessive retinopathy
-
Leber congenital amaurosis 2 3 trials
-
Retinitis pigmentosa 20 0 trials
-
Can a One-Time gene therapy preserve sight for years? a 15-Year study aims to find out
Disease control OngoingThis study follows people who previously received a one-time gene therapy injection for a rare inherited eye disease caused by RPE65 mutations. Researchers will test vision and light sensitivity over up to 15 years to see how well the treatment holds up. The goal is to understand…
Sponsor: Genentech, Inc. • Aim: Disease control
Last updated Aug 21, 2026 00:00 UTC
-
One-Time gene injection aims to restore sight in rare eye disease
Disease control OngoingThis phase 3 trial tests a gene therapy called LX101 in 30 people with inherited retinal dystrophy caused by RPE65 mutations. Participants receive a single injection of LX101 into the eye, while a control group gets no treatment. The study measures changes in functional vision an…
Phase 3 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
-
Experimental gene therapy aims to restore sight in rare eye disease
Disease control OngoingThis early study tests a gene therapy called LX101 in 9 people with a rare inherited eye disease caused by RPE65 gene mutations. The therapy delivers a working copy of the gene into the retina via a single injection. The main goals are to check safety and see if it can improve vi…
Phase 1 • Sponsor: Innostellar Biotherapeutics Co.,Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC